TIDMDNL
RNS Number : 8385E
Diurnal Group PLC
12 July 2021
--12 July 2021
Diurnal Group plc
("Diurnal" or the "Company")
Diurnal receives agreement from US FDA on Special Protocol
Assessment for Chronocort(R)
Pivotal Phase 3 trial expected to commence in the US in H2
2021
Diurnal Group plc (AIM: DNL), the specialty pharmaceutical
company targeting patient needs in chronic endocrine (hormonal)
diseases, today announces that the US Food and Drug Administration
(FDA) has agreed a Special Protocol Assessment (SPA) for
Chronocort(R) (modified-release hydrocortisone) for the design,
endpoints and statistical analysis approach of a pivotal study of
Chronocort(R) for the treatment for Congenital Adrenal Hyperplasia
(CAH) in the US. The SPA provides agreement that the Phase 3 trial
design adequately addresses objectives that would support the
regulatory submission for drug approval.
The pivotal study is entitled "A randomised, double-blind,
active-controlled, Phase 3 study of Chronocort(R) compared with
immediate-release hydrocortisone replacement therapy in
participants aged 16-years and over with Congenital Adrenal
Hyperplasia (CAH)" - CONnECT - and is anticipated to start during
H2 2021. Study start-up activities have already been commenced by
the Company to enable a timely initiation and recruitment of up to
150 subjects with CAH who will be treated for a period of 52
weeks.
Chronocort(R) is a preparation of hydrocortisone that has been
specifically designed for patients with CAH, an orphan condition,
caused by deficiency of adrenal enzymes, most commonly
21-hydroxylase. Diurnal holds the relevant Orphan Drug Designation
for use of Chronocort(R) in CAH in the US. Approximately two-thirds
of CAH patients are estimated to have poor disease control, leading
to elevated androgen levels. The condition is estimated to affect a
total of approximately 16,000 patients in the US.
Martin Whitaker, Chief Executive Officer of Diurnal,
commented:
"We are pleased to have concluded a series of positive
discussions with the FDA around the design of our pivotal
Chronocort(R) study to support a regulatory submission for
marketing approval in the US. During the first half of 2021 the
Diurnal team has been working hard to complete the required
preparation activities for this trial and we look forward to
starting this study as soon as possible."
This is a business press release containing financial
information and/or data for the benefit of shareholders and
potential investors. Data are included to allow informed investment
decisions.
This announcement contains inside information for the purposes
of Article 7 of Regulation (EU) 596/2014 (MAR).
For further information, please visit www.diurnal.co.uk or
contact:
+44 (0)20 3727
Diurnal Group plc 1000
Martin Whitaker, Chief Executive Officer
Richard Bungay, Chief Financial Officer
Panmure Gordon (UK) Limited (Nominated Adviser +44 (0)20 7886
and Sole Broker) 2500
Corporate Finance: Freddy Crossley, Emma Earl
Corporate Broking: Rupert Dearden
+44 (0)20 3727
FTI Consulting (Media and Investor Relations) 1000
Simon Conway
Victoria Foster Mitchell
Alex Davis
Notes to Editors
About the FDA's Special Protocol Assistance
The SPA process documents the FDA's official evaluation and
written guidance on the design and size of proposed protocols and
agreement that the design and planned analysis of a study can
adequately address objectives in support of a regulatory
submission. Final determinations for marketing application
approval, however, are made after a complete review of a marketing
application and are based on the entire data in the application.
The agreed SPA may only be changed through a written agreement
between the sponsor and the FDA, or if the FDA becomes aware of a
substantial scientific issue essential to product efficacy or
safety.
About Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is a rare condition caused
by deficiency of adrenal enzymes, most commonly 21-hydroxylase.
This enzyme is required to produce the adrenal steroid hormone,
cortisol. The block in the cortisol production pathway causes the
over-production of male steroid hormones (androgens), which are
precursors to cortisol. The condition is congenital (inherited at
birth) and affects both sexes. The cortisol deficiency and
over-production of male sex hormones can lead to increased
mortality, infertility and issues during sexual development
including ambiguous genitalia, premature (precocious) sexual
development and short stature. Sufferers, even if treated, remain
at risk of death through an adrenal crisis.
Current therapy for CAH uses a variety of generic glucocorticoid
(steroids including hydrocortisone, dexamethasone, prednisolone and
prednisone in the US) with no standard treatment regimen.
Approximately two-thirds of CAH patients are estimated to have poor
disease control, leading to elevated androgen levels. The condition
is estimated to affect approximately 16,000 patients in the US,
with over 400,000 in the rest of the world.
About Chronocort(R) (modified-release hydrocortisone)
Chronocort(R) is a preparation of hydrocortisone that has been
specifically designed to mimic the circadian rhythm of cortisol
when given in a twice-a-day "toothbrush" regimen (administered last
thing at night before sleep and first thing in the morning on
waking) to control androgen excess and chronic fatigue in patients
with diseases of cortisol deficiency. The first planned indication
for Chronocort(R) in the US is Congenital Adrenal Hyperplasia (CAH)
in adults and children older than 16 years of age. Chronocort(R)
has been extensively studied in 239 human subjects including 138
CAH patients who have taken part in clinical trials in Europe and
the US.
Although Chronocort(R) is still an investigational product in
the US, Chronocort(R) (known in Europe as Efmody(R) ) has already
achieved marketing authorisation approval from the European
Commission (in the European Economic Area) and the Medicines and
Healthcare products Regulatory Agency (in Great Britain). These
marketing authorisations for Efmody(R) are based on a Phase 3 study
conducted in a total of 122 patients enrolled across 11 clinical
sites, including a site in the US, the largest ever interventional
clinical trial completed in CAH. The Phase 3 data was supported by
detailed analysis of data from an open-label safety extension study
for patients completing treatment in the Phase 3 study, which is
assessing the impact of treatment with Efmody(R) over an extended
period, with a number of patients on this trial having been treated
for over 54 months. Patient retention rates in this study have been
high and patients on this trial have, to date, shown sustained
benefit from extended Efmody(R) treatment.
The human medicine European public assessment report (EPAR) for
Efmody(R) can be viewed here and the Summary of Product
Characteristics (SmPC) here .
About Diurnal Group plc
Diurnal Group plc is a European, UK-headquartered, specialty
pharmaceutical company dedicated to developing hormone therapeutics
to aid lifelong treatment for rare and chronic endocrine
conditions, including congenital adrenal hyperplasia, adrenal
insufficiency, hypogonadism and hypothyroidism. Its expertise and
innovative research activities focus on circadian-based
endocrinology to yield novel product candidates in the rare and
chronic endocrine disease arena.
For further information about Diurnal, please visit
www.diurnal.co.uk
Date of Preparation: July 2021 Code: CORP-GB-0141
This information is provided by RNS, the news service of the
London Stock Exchange. RNS is approved by the Financial Conduct
Authority to act as a Primary Information Provider in the United
Kingdom. Terms and conditions relating to the use and distribution
of this information may apply. For further information, please
contact rns@lseg.com or visit www.rns.com.
RNS may use your IP address to confirm compliance with the terms
and conditions, to analyse how you engage with the information
contained in this communication, and to share such analysis on an
anonymised basis with others as part of our commercial services.
For further information about how RNS and the London Stock Exchange
use the personal data you provide us, please see our Privacy
Policy.
END
AGRUAORRABUBAUR
(END) Dow Jones Newswires
July 12, 2021 02:00 ET (06:00 GMT)
Diurnal (LSE:DNL)
Gráfica de Acción Histórica
De Mar 2024 a Abr 2024
Diurnal (LSE:DNL)
Gráfica de Acción Histórica
De Abr 2023 a Abr 2024
